Docs biased against research from poor countries, study finds; Astellas probed over patient-assistance charity support; Acorda reports deaths in Parkinson's trial
AstraZeneca sales fall on generic competition; Anthem broadens access for Duchenne muscular dystrophy drug; researchers are developing new biopsy techniques
The association said it is preparing for new FDA approvals for therapies that treat central nervous system and muscular system diseases.
Nonprofits receive FDA approval for Chagas drug; Gilead's buyout of Kite will put it back in pricing fray; most cancer patients don't receive fertility info
Biogen reports better-than-expected Spinraza sales; FDA to discuss opioid prescriptions with payers; UnitedHealth Group launches media review
About 4.4 billion prescriptions filled in the U.S. in 2016; Sanofi and Ablynx reach I-O deal; biotech M&A slows in H1
AstraZeneca CEO reportedly moving to Teva; the FDA reverses on approval for Amicus drug; Merck names winners of Alexa challenge
This includes Sarepta Therapeutics' Exondys 5 and Vertex Pharmaceuticals' Orkambi.
Intarcia submits diabetes drug-delivery pump for FDA approval; Shire to build rare-disease innovation hub; AstraZeneca to resume durvalumab trials
Bayer submits Stivarga to FDA for liver-cancer indication; emails show FDA's divisiveness over Duchenne drug approval; insurers want new president to address ACA financial risks
Data analytics, relationship building, and improved patient support are the keys to differentiating your rare-disease drug brands, according to these four marketers.
The agency announced a partnership with Vencore Health Analytics to help pair rare disease patients with drugmakers.
The biotech company is working with the foundation and Brown University to develop new regimens for the socially stigmatized condition.
The FDA declines to approve two drugs for Duchenne muscular dystrophy; government watchdog questions FDA's post-market drug tracking; WebMD denies acquisition talks
The National Organization for Rare Disorders and the University of Maryland will develop a training program to help caregivers and patients participate in research.
Amgen's PCSK9 inhibitor is the second in its class to get an FDA panel endorsement; a new drug could threaten Shire's hold on a rare-disease category; a study shows pharma invests less in Hispanic audiences than in other groups
Nexium tops the list of Medicare's spending on brand drugs; Gilead Sciences doubles its first-quarter profit; Pfizer reportedly wants to buy a rare-disease drug company
The cancer and immunology drug may help patients with a condition that affects two people in every one million.
Horizon Pharma's purchase of Hyperion Therapeutics will add two rare-disease drugs to its portfolio.
The duo hope to begin testing a new treatment within three years.
The $38-million deal expands the reach of rare-disease campaigns.
Industry rare disease awareness efforts included a pipeline overview at Pfizer as well as BI's efforts to draw attention to IPF.
The FDA approved NPS Pharmaceuticals's rare-disease drug Friday. The company, which Shire shelled out $5.2 billion to buy, expects it to hit the market during the second-quarter.
The specialty biopharma firm says it's hiring new commercial managers after a buying spree.
BioMarin to acquire Dutch biotech Prosensa; Woodcock says significant efficacy key to Breakthrough Therapy designation; Stryker considers $16 billion bid for UK devicemaker Smith and Nephew.
Success today is predicated on recognizing that every patient is different, every disease is different, every drug, every breakthrough and every cure is different.
The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
FDA inspectors have cited the manufacturer of the rare-disease drug Soliris (eculizumab) for poor manufacturing practices.
The oral medication Cerdelga joins Cerezyme in its Gaucher disease arsenal. Sanofi expects the drug will be priced "on par" with Cerezyme, which goes for around $300,000 a year in the US.
Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.